Stoke Therapeutics, Inc.

Stoke Therapeutics, Inc. is a biotechnology company dedicated to developing RNA-based medicines to address the underlying causes of severe genetic diseases by precisely upregulating protein expression. Founded in 2014 and headquartered in Bedford, Massachusetts, with additional offices in Cambridge, Massachusetts, the company aims to restore target proteins to near normal levels.

The company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to design antisense oligonucleotides (ASOs) that selectively restore protein levels. Its lead clinical candidate, zorevunersen (STK-001), is currently in Phase 3 clinical trials for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke's pipeline also includes STK-002 for Autosomal Dominant Optic Atrophy (ADOA), which is in Phase 1 clinical trials or preclinical stages, and earlier-stage programs for SYNGAP-1 disorders and Rett syndrome. The company's initial focus is on haploinsufficiency diseases of the central nervous system and the eye.

Stoke Therapeutics is a publicly traded company on NASDAQ under the ticker symbol STOK. Ian F. Smith serves as the Chief Executive Officer, having been appointed in October 2025. Recent developments include the appointment of Clare Kahn, Ph.D., to its Board of Directors in April 2026. The company anticipates completing enrollment for its Phase 3 EMPEROR study for zorevunersen in Q2 2026, with a data readout expected in mid-2027 and a rolling New Drug Application (NDA) submission planned for the first half of 2027 under Breakthrough Therapy designation. Stoke has also established collaborations with companies like Biogen and Acadia Pharmaceuticals.